Myelodysplastic Syndromes | Niche and Rare Pharmacor | G7 | 2015

Myelodysplastic syndromes (MDS) consists of a group of hematological stem cell disorders that are heterogeneous in cause and manifestations but share the common features of aberrant hematopoiesis and deteriorating cytopenias. Patients with MDS most commonly display anemia, but suffer from additional cytopenias as well, with outcomes such as extreme fatigue, high rates of infections, excessive bleeding, and shortened life span. Lower-risk MDS patients are typically treated for anemia and associated issues, while higher-risk MDS patients are treated more aggressively in attempt to eliminate the clonal population and extend lifespan.

This report provides an overview of the MDS landscape featuring a comprehensive analysis of patient populations, current therapies and medical practices, and opportunities for emerging therapies. The report identifies a very high level of unmet need for new, disease-modifying MDS therapies and provides expert insight on potential drug targets. At this time, the only potentially curative therapy for MDS is allogeneic hematopoietic stem cell transplant (HSCT). However, interviewed experts report that approximately 90% of the MDS patient population is ineligible for HSCT owing to advanced age, difficulty finding a donor, or most commonly, poor health status. Current treatment options for MDS are very limited and frequently ineffective. They include lenalidomide (Revlimid) and erythropoiesis-stimulating agents (ESAs) for lower-risk patients, and the hypomethylating agents azacitidine (Vidaza) and decitabine (Dacogen) for higher-risk patients. Nearly half of patients using these approved therapies are refractory or become unresponsive, and there are no approved second-line therapies for MDS.